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Improvements in Mucopolysaccharidosis I Mice After Adult Retroviral Vector–mediated Gene Therapy with Immunomodulation

20 Feb 2007

Abstract excerpt

Mucopolysaccharidosis I (MPS I) is caused by deficient alpha-L-iduronidase (IDUA) activity and results in the accumulation of glycosaminoglycans and multisystemic disease. Gene therapy could program cells to secrete mannose 6-phosphate-modified IDUA, and enzyme in blood could be taken up by other cells. Neonatal retroviral vector (RV)-mediated gene therapy has been shown to reduce the manifestations of murine MPS...

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PMID: 17311010
DOI: 10.1038/sj.mt.6300112

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